CA2996280A1 - Oligomere antisens pour outrepasser exon 45 - Google Patents
Oligomere antisens pour outrepasser exon 45 Download PDFInfo
- Publication number
- CA2996280A1 CA2996280A1 CA2996280A CA2996280A CA2996280A1 CA 2996280 A1 CA2996280 A1 CA 2996280A1 CA 2996280 A CA2996280 A CA 2996280A CA 2996280 A CA2996280 A CA 2996280A CA 2996280 A1 CA2996280 A1 CA 2996280A1
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- Prior art keywords
- oligomer
- nucleotide sequence
- pharmaceutically acceptable
- hydrate
- acceptable salt
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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- 125000000999 tert-butyl group Chemical group [H]C([H])([H])C(*)(C([H])([H])[H])C([H])([H])[H] 0.000 description 1
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- QEMXHQIAXOOASZ-UHFFFAOYSA-N tetramethylammonium Chemical class C[N+](C)(C)C QEMXHQIAXOOASZ-UHFFFAOYSA-N 0.000 description 1
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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Abstract
La présente invention concerne un oligomère qui permet le saut de l'exon 45 dans le gène humain de la dystrophine.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2015182145 | 2015-09-15 | ||
| JP2015-182145 | 2015-09-15 | ||
| PCT/JP2016/077305 WO2017047707A1 (fr) | 2015-09-15 | 2016-09-15 | Acide nucléique antisens |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CA2996280A1 true CA2996280A1 (fr) | 2017-03-23 |
| CA2996280C CA2996280C (fr) | 2024-05-07 |
Family
ID=58289325
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| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2996280A Active CA2996280C (fr) | 2015-09-15 | 2016-09-15 | Oligomere antisens pour outrepasser exon 45 |
Country Status (28)
| Country | Link |
|---|---|
| US (3) | US10144931B2 (fr) |
| EP (2) | EP3351633B1 (fr) |
| JP (4) | JP6384845B2 (fr) |
| KR (3) | KR20220053048A (fr) |
| CN (3) | CN108026531B (fr) |
| AU (1) | AU2016324800B2 (fr) |
| CA (1) | CA2996280C (fr) |
| CO (1) | CO2018002557A2 (fr) |
| CY (1) | CY1123119T1 (fr) |
| DK (1) | DK3351633T3 (fr) |
| ES (1) | ES2808049T3 (fr) |
| HR (1) | HRP20201125T1 (fr) |
| HU (1) | HUE050061T2 (fr) |
| IL (1) | IL258065B (fr) |
| LT (1) | LT3351633T (fr) |
| MX (1) | MX2018002955A (fr) |
| MY (1) | MY185390A (fr) |
| PH (1) | PH12018500568A1 (fr) |
| PL (1) | PL3351633T3 (fr) |
| PT (1) | PT3351633T (fr) |
| RS (1) | RS60493B1 (fr) |
| RU (1) | RU2724554C2 (fr) |
| SG (1) | SG11201802138TA (fr) |
| SI (1) | SI3351633T1 (fr) |
| TW (1) | TWI725990B (fr) |
| UA (1) | UA123359C2 (fr) |
| WO (1) | WO2017047707A1 (fr) |
| ZA (1) | ZA201801682B (fr) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
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| CN108026531B (zh) * | 2015-09-15 | 2021-09-14 | 日本新药株式会社 | 反义核酸 |
| IL295755A (en) | 2015-10-09 | 2022-10-01 | Wave Life Sciences Ltd | Oligonucleotide preparations and methods thereof |
| MX2021001281A (es) | 2018-08-02 | 2021-07-15 | Dyne Therapeutics Inc | Complejos dirigidos a músculos y sus usos para el tratamiento de distrofinopatías. |
| CA3108289A1 (fr) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Complexes de ciblage musculaire et leurs utilisations pour le traitement de la dystrophie musculaire facio-scapulo-humerale |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| GB201821269D0 (en) * | 2018-12-28 | 2019-02-13 | Nippon Shinyaku Co Ltd | Myostatin signal inhibitor |
| US20230193282A1 (en) | 2021-04-30 | 2023-06-22 | Sarepta Therapeutics, Inc. | Treatment methods for muscular dystrophy |
| EP4361269A1 (fr) * | 2021-06-23 | 2024-05-01 | Nippon Shinyaku Co., Ltd. | Combinaison d'oligomères antisens |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| CN118488959A (zh) | 2021-12-27 | 2024-08-13 | 日本新药株式会社 | 低聚核酸化合物的制造方法 |
| WO2023168427A1 (fr) | 2022-03-03 | 2023-09-07 | Yale University | Compositions et procédés d'administration de polynucléotides thérapeutiques pour saut d'exon |
| IL315280A (en) | 2022-03-17 | 2024-10-01 | Sarepta Therapeutics Inc | Morpholino phosphorodiamidate oligomer conjugates |
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| US2567664A (en) | 1948-04-22 | 1951-09-11 | Willis G Ewell | Chicken culling device |
| AU655164B2 (en) | 1989-12-20 | 1994-12-08 | Antivirals Inc. | Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages |
| JP2924179B2 (ja) | 1993-02-19 | 1999-07-26 | 日本新薬株式会社 | グリセロール誘導体，デバイス及び医薬組成物 |
| JP2000325085A (ja) * | 1999-05-21 | 2000-11-28 | Masafumi Matsuo | デュシェンヌ型筋ジストロフィー治療剤 |
| US6727355B2 (en) * | 2000-08-25 | 2004-04-27 | Jcr Pharmaceuticals Co., Ltd. | Pharmaceutical composition for treatment of Duchenne muscular dystrophy |
| JP4836366B2 (ja) * | 2000-08-25 | 2011-12-14 | 雅文 松尾 | デュシェンヌ型筋ジストロフィー治療剤 |
| EP1191097A1 (fr) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction du "exon-skipping" dans des cellules eukaryotes |
| ES2554660T3 (es) | 2002-11-25 | 2015-12-22 | Masafumi Matsuo | Fármacos de ácido nucleico ENA que modifican el corte y empalme en precursores de ARNm |
| WO2004083432A1 (fr) | 2003-03-21 | 2004-09-30 | Academisch Ziekenhuis Leiden | Modulation de la reconnaissance d'exons dans le pre-arnm par interference avec la structure d'arn secondaire |
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